HIT-CF program for rare mutations – August 2023 Update HCFA TEAM October 30, 2023

HIT-CF program for rare mutations – August 2023 Update

Resubmission of the “Choices” clinical trial to CTIS, after its initial rejection, is being prepared by the European HIT-CF Europe project team, giving promising signals for the start of the trial at the beginning of the new year.

Individuals selected for the trial at participating European centres have already been notified and HIT-CF is negotiating with other partners to set up trials for those unable to participate in CHOICES. A total of 52 patients, selected from European centres, will be enrolled in the trial, which will examine the effect of a triple combination of CFTR modifiers on increasing the number of functional CFTR channels on the cell surface. In addition, at the same time as the study medicines were being tested , Tezacaftor/Ivacaftor (Symkevi) treatment was also tested in organoids and the individualised response of organoids to Symkevi has been disclosed to HIT-CF participants not eligible for the Choices study.

Read more about the European programme and its results in the HIT-CF newsletter for August 2023 here.

More information about the HIT-CF Europe programme can be found here.

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